The firm said it has received positive feedback from the FDA on the design of a Phase III trial of ELI-002 in KRAS-mutant pancreatic cancer.
Their study showed that DCIS patients with pathogenic variants in BRCA1/2 and PALB2 had a higher risk of developing invasive breast cancer than those without.
The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.
BH-30643 is an OMNI-EGFR inhibitor that targets classical, atypical, and compound EGFR mutations and acquired resistance mutations in HER2.
The companies will focus on advancing AXV-101, with which they hope to treat retinal dystrophy by targeting mutations in the BBS1 gene.
In a Phase I/II trial, patients with X-linked chronic granulomatous disease will receive a treatment created using a new approach to base editing.
Doctors and patient advocates supporting pretreatment testing at times challenged FDA and other experts who aren't swayed by available evidence.
The deal advances Galapagos' plan to develop a decentralized CAR T-cell therapy for non-Hodgkin lymphoma with a one-week vein-to-vein time.